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slow the progress of Parkinson’s. (There is anecdotal evidence that smokers don’t suffer the neurodegeneration of Parkinson’s as much as non-smokers.) The second trial, run by University College, London, is examining the possibility that exenatide (Byetta), a type 2 diabetes drug sold by AstraZeneca (NYSE: AZ), can also slow or halt the disease.
Both trials have funding from the nonprofit Michael J. Fox Foundation. Both treatments are being repurposed by investigators, so if the Phase 2 studies are successful, it’s not clear who will pay for expensive Phase 3 trials or a commercial effort with less-than-appealing intellectual property rights attached to the drugs. The Fox Foundation’s senior vice president of research programs Brian Fiske declined to comment about commercial prospects. “We’re focused on building the data around the drugs,” he said. But deep-pocketed philanthropists are pushing more cash into biomedical research and treatments. Could the upcoming Parkinson’s data become a test case for nonprofit funding of a major drug to market?
Disease Area: Postpartum Depression
Company: Sage Therapeutics
Trial: Phase 3
Data Expected: Late 2017
Why we’re watching: Postpartum depression strikes women after childbirth, often severely, and affects 500,000 to 750,000 mothers in the U.S. every year. Yet there are no drugs approved for it. SAGE-547—which is thought to reverse the rapid decrease in the brain chemical allopregnanolone—has a chance to be the first. But Sage (NASDAQ: SAGE) must show that the early promising signs in a small Phase 2 study weren’t a mirage.
That’s a big test, because time and again drugs for depression have shown early promise only to fizzle in late-stage trials, often due to a higher than expected placebo response. Sage’s Phase 2 trial only included 21 women; 10 of them got SAGE-547 and 11 a placebo. Seven of the 10 women on SAGE-547 were free from depression 30 days after taking the drug, compared to two of the 11 on placebo. Sage is enrolling as many as 220 women in its Phase 3 trial.
Disease area: Transthyretin amyloidosis
Data expected: Mid-2017
Why we’re watching: This Phase 3 trial of Alnylam Pharmaceuticals’ patisiran is important for two reasons. First, it could support the first-ever FDA approval filing for an RNA interference (RNAi) drug, a type of medicine that knocks down or “silences” genes before they produce harmful proteins. The discoverers of RNAi won a Nobel Prize a decade ago. Soon after, drugs based on the premise were the subject of massive pharma deals. (Many included Alnylam.)
The frenzy faded into near-oblivion after several years. Fighting through setbacks, Alnylam (NASDAQ: ALNY) has rebuilt a $3.5 billion market capitalization. But success remains elusive. It has already ditched a drug in Phase 3 because of safety problems, and after patisiran its most advanced is fitusiran for hemophilia, in Phase 2. Alnylam has already built a commercial team for patisiran. Success could bring to market a treatment for a form of the rare disease transthyretin amyloidosis that causes damage to the nervous system. Failure would raise a new set of questions about RNAi’s potential—and about Alnylam’s future.
Disease area: Zika virus
Trial: Phase 1
Data expected: Mid to late 2017
Why we’re watching: Zika vaulted from obscurity into the headlines in 2016 because of its link to birth defects such as microcephaly and its spread north from Brazil. Several efforts to develop vaccines against the virus are underway. Plymouth Meeting, PA-based Inovio Pharmaceuticals (NASDAQ: INO) was the first into clinical trials, using financial backing from South Korea’s GeneOne Life Science. Forty subjects have had “robust immune responses” with the GLS-5700 vaccine so far with “no significant safety concerns” after 14 weeks, the company said this week. That study is scheduled for completion in November 2017. A second Phase 1 trial, enrolling 160 patients in Puerto Rico where Zika has spread, could also report data next year.
Being first out of the gate is no guarantee that Inovio’s vaccine will be the first to cross the finish line. The National Institutes of Health is sponsoring or collaborating on at least eight Zika trials. Other industry developers in the ring include the vaccines division of Sanofi (NYSE: SNY), working with the Walter Reed Army Institute of Research and the NIH; Cambridge, MA-based Moderna Therapeutics, which is waiting for a clinical go-ahead from the FDA; and Japan’s Takeda Pharmaceutical, which has a $19.8 million U.S. defense grant to bring a Zika vaccine through Phase 1 clinical trials. That funding could reach $312 million if a vaccine advances far enough to file for regulatory approval.